title: Guidance on preparing an investigational new drug application for fecal microbiota transplantation studies.
abstract: Fecal microbiota transplantation (FMT) is an effective treatment for Clostridium difficile infections that are refractory to antibiotic therapy. Because of the important roles of the microbiota in the function of the gastrointestinal tract and other aspects of human physiology, there is a growing interest in studying FMT for other clinical indications. The US Food and Drug Administration regulates clinical studies to evaluate the safety and efficacy of FMT. Studies of FMT for recurrent Clostridium difficile infection or other indications could require submission of an investigational new drug application. Most academic physicians and investigators do not have the regulatory experience necessary to undertake this process. We provide guidance to researchers on the preparation and submission of investigational new drug applications to study FMT.
authors.affiliation: Women's Medicine Collaborative, The Miriam Hospital, Brown University Alpert School of Medicine, Providence, Rhode Island. Electronic address: colleen_r_kelly@brown.edu.
authors.forename: Colleen R
authors.initials: CR
authors.lastname: Kelly
authors.affiliation: Helen DeVos Children's Hospital, Michigan State University, Grand Rapids, Michigan.
authors.forename: Sachin S
authors.initials: SS
authors.lastname: Kunde
authors.affiliation: Department of Medicine, University of Minnesota, Minneapolis, Minnesota.
authors.forename: Alexander
authors.initials: A
authors.lastname: Khoruts
mesh_terms: D001691:Biological Therapy / Q000379:methods*; D002985:Clinical Protocols; D016360:Clostridioides difficile; D004761:Enterocolitis, Pseudomembranous; D005243:Feces / Q000382:microbiology*; D006801:Humans; D007422:Intestines / Q000382:microbiology; D017387:Investigational New Drug Application / Q000458:organization & administration; D064307:Microbiota; D012008:Recurrence; D013048:Specimen Handling; D014180:Transplantation / Q000379:methods; D014481:United States; D014486:United States Food and Drug Administration
keywords: AE; CBER; CDI; CRF; Center for Biologics Evaluation and Research; Clostridium difficile infection; FDA; FMT; Food and Drug Administration; HIV; IB; IBD; IC; IND; IRB; R-CDI; SAE; adverse event; case report form; fecal microbiota transplantation; human immunodeficiency virus; inflammatory bowel disease; informed consent; institutional review board; investigational new drug; investigator's brochure; recurrent CDI; serious adverse event